New perspectives for the treatment of autoimmune diseases

When two rare autoimmune disorders – immune thrombocytopenia (ITP) and antiphospholipid syndrome (APS) – combine in one patient, it can lead to a life-threatening spiral of severe hemorrhaging and blood clotting. An interdisciplinary team from the Medical Clinic and Polyclinic III at LMU University Hospital has demonstrated for the first time that a novel form of immunotherapy with so-called bispecific antibodies can silence the underlying autoimmune response in a targeted manner. The findings of this groundbreaking treatment have now been published in the New England Journal of Medicine.

In immune thrombocytopenia, the immune system turns against its own platelets, resulting in spontaneous bleeding. Antiphospholipid syndrome, by contrast, leads to pathogenic coagulation activation and life-threatening thromboses. If both disorders coincide, a dangerous contradiction arises: The body threatens to hemorrhage and to form blood clots at the same time. “Such patients present us with extreme medical challenges,” explains Professor Karsten Spiekermann, hemostaseologist at LMU University Hospital.

Novel immunotherapy activates body’s T cells

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In recent years, a special form of immunotherapy known as CAR T-cell therapy has had impressive successes in treating autoimmune disorders. However, this therapy is complex to produce for individual patients and requires chemotherapy, which can cause infertility and new cancers.

The Munich-based team led by Professor Marion Subklewe, Dr. Adrian Gottschlich, Professor Karsten Spiekermann, and Professor Michael von Bergwelt leveraged its expertise in T-cell-recruiting immunotherapies to test an alternative, gentler method: the bispecific antibody blinatumomab. “Bispecific antibodies work like a molecular connector between T cells and pathogenic B cells,” explains Marion Subklewe, head of the Immunotherapy focus area at LMU University Hospital. “In this way, we can selectively silence the cells that produce harmful autoantibodies – without any chemotherapy.”

Disappearance of autoantibodies – stable platelets

As part of a compassionate-use program, a young patient with therapy-resistant ITP and APS received two cycles of treatment with blinatumomab. Shortly after therapy initiation, her platelet count increased and the pathogenic antibodies disappeared completely. “After countless unsuccessful therapeutic attempts, we were able to gradually reduce the blood-forming drugs for the first time – while the platelets remained stable,” reports Adrian Gottschlich, lead author of the study. “At the same time, the autoantibodies that were responsible for both the bleeding tendency and the thromboses disappeared.” Accompanying laboratory analyses, carried out in collaboration with PD Dr. Rainer Kaiser (Medical Clinic I), showed moreover that platelet aggregation fully normalized after the therapy.

Since the treatment, the patient has normal platelet counts and is free from pain, hemorrhaging, and thromboses. For the first time in years, she has been able to resume oral blood-thinning therapy without daily subcutaneous injections. This represents a huge gain in quality of life and a return to normal life. “The data show the tremendous potential of targeted immunotherapies for autoimmune disorders,” emphasizes Michael von Bergwelt. “Bispecific antibodies in particular are opening up safe new therapy options, especially for young patients.”

Adrian Gottschlich et al.: Blinatumomab in Combined Immune Thrombocytopenia and Antiphospholipid Syndrome. The New England Journal of Medicine 2026